Limited coverage drugs – sapropterin dihydrochloride

Last updated on September 27, 2024

Generic name

sapropterin dihydrochloride

Strength/Form

100 mg tablet

100 mg and 500 mg sachet with powder for oral solution

Special Authority criteria

Approval period

Initial coverage

For the treatment of hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-responsive (BH4-responsive) phenylketonuria (PKU) in patients who meet ALL of the following initiation criteria:

1. a confirmed diagnosis of phenylketonuria (PKU) AND managed by a physician specialized in metabolic/biochemical diseases

2. adherence to low-protein diet and formulas AND baseline protein intake assessment by a dietitian

3. baseline blood phenylalanine (Phe) levels > 360 µmol/L despite adherence to a low-protein diet (requires at least 2 baseline levels during the preceding  3- to 6-month time frame)

4. ability to adhere to medication regimen

6 months

Renewal coverage

For the treatment of HPA due to BH4-responsive PKU in patients who meet ALL of the following renewal criteria:

  1. adherence to low-protein diet and formulas and sapropterin, AND followed by a dietitian.
  2. Phe levels meet treatment targets (at least 2 levels measured at least 1 month apart):
    1. normal sustained blood Phe levels < 360 μmol/L OR
    2. sustained blood Phe reduction of at least 30% compared to baseline if the Phe baseline level was < 1200 μmol/L OR
    3. sustained blood Phe reduction of at least 50% compared to baseline if the Phe baseline level was > 1200 μmol/L
  3. demonstrated increase of dietary protein tolerance based on targets set between the clinician and patient AND managed by a physician specialized in metabolic/biochemical diseases.

1 year

Pregnant patients may qualify for coverage upon meeting the following modified criteria:

1. diagnosis of phenylketonuria (PKU) confirmed through an approved test and managed by a physician specialized in metabolic/biochemical diseases.

2. baseline blood phenylalanine (Phe) levels > 360 µmol/L despite adherence with all recommendations for dietary intervention and monitoring. Pregnant patients only require demonstration of one baseline Phe level > 360 µmol/L despite compliance with low-protein diet.

Note: Pregnant patients will be eligible for funding for the duration of pregnancy. Renewal of funding will require meeting the criteria for non-pregnant patients.

1 year

Practitioner exemptions

  • None

Special notes

  • A maximum of 20 mg/kg per day will be approved for eligible patients.

Special Authority requests